GeneticsbioRxivPreprint
Base editing as a genetic treatment for spinal muscular atrophy
Christiano R. R. Alves, Leillani L. Ha, Rebecca Yaworski…·Benjamin P. Kleinstiver
This paper describes the optimization of base editing to correct the SMN2 exon 7 mutation in SMA, achieving high editing efficiency and increased SMN protein levels in vitro and in vivo.
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